MATRIX-RARE Background/study objectives

Personalized treatment based on molecular characteristics of the patient’s tumour has changed the treatment of some cancers, and has given hope to many patients. Several ongoing clinical studies are investigating the concept of re-purposing drugs to patients with advanced cancers who have progressed on standard treatment. We see that some of these patients have durable responses and clinical benefit. However, it is known that tumour cells are more susceptible to therapy in earlier treatment lines, and it is therefore likely that patients would benefit more if effective treatment was tried earlier in the disease course. This may be especially important for rare aggressive cancers, for which standard treatments are few and where the rapid course often makes it too late to include the patient in a clinical trial when progression is first observed.

In the MATRIX-RARE study, patients with advanced rare aggressive cancers undergo molecular screening and receive personalized biomarker-based therapy at diagnosis / before receiving all available lines of treatments. The study will evaluate the toxicity and efficacy of the drugs included. Mechanisms of primary and acquired resistance will be studied by the use of liquid biopsies and serial fresh tumour biopsies for WGS. Other exploratory objectives include long-term overall survival, assessments of quality of life and development of imaging artificial intelligence (AI) methods for response evaluation.